PB2428: CLINICAL EFFICACY AND HEALTH-RELATED QUALITY OF LIFE IN SEVERE APLASTIC ANEMIA TREATED BY UMBILICAL CORD BLOOD TRANSPLANTATION: A CROSS-SECTIONAL SURVEY IN CHINA
Authors: Ping LI, Jun Peng, Zengjun LI
Journal: HemaSphere (2023)

Topic: 22. Stem cell transplantation - Clinical Background: One of the main pathogenesis of AA is the deficiency of cytoplasm and number of hematopoietic stem/progenitor cells. Hematopoietic stem cell transplantation is expected to solve this problem fundamentally. Due to the limitation of allogeneic hematopoietic stem cell source and GVHD produced after transplantation, the therapeutic effect is significantly affected. So the search for new treatments has become critical. In recent years, cord blood, as the third source of hematopoietic stem cells besides bone marrow and peripheral blood, has been playing an increasingly important role in the field of hematopoietic stem cell transplantation because of its good performance in multidisciplinary diseases such as blood system. Aims: To compare the clinical efficacy of umbilical cord blood transplantation, bone marrow/peripheral blood allogeneic hematopoietic stem cell transplantation and immunosuppression treatment in severe aplastic anemia from the perspective of quality of life, and to explore the application of umbilical cord blood transplantation in severe aplastic anemia. Methods: 112 cases of severe aplastic anemia were selected.According to the treatment methods, the patients were divided into five groups: umbilical cord blood transplantation group (UCBT), bone marrow/peripheral allogeneic hematopoietic stem cell transplantation group (BM/PB Allo-HSCT), ATG/ALG + CsA group, CsA ± androgen group and supportive treatment group. The clinical efficacy was statistically analyzed.The Medical Outcomes Study 36-item short-form health survey (SF-36) was used to compare quality of life among treatment groups. 172 healthy, sex-and age-matched subjects were used as controls. Results: After 54 (3-164) months of follow-up, the median age at treatment in the UCBT group was 18[3-48] years, the overall survival rate was 92.8572% (26/28), the median time for neutrophil hematopoietic reconstruction was 32 (11-124) d, and the median time for platelet hematopoietic reconstruction was 57 (10-365) d. The hematopoietic reconstruction rate was 89.285% (25/28). In the BM/PB Allo-HSCT group, the median age at treatment was 23[6-48] years, the overall survival rate was 82.0513% (32/39), the median time for neutrophil hematopoietic reconstruction was 13 (9-58) d, and the median time for platelet hematopoietic reconstruction was 13 (5-95) d. The hematopoietic reconstruction rate was 82.051% (32/39). The time required for hematopoietic reconstruction was significantly different between the two groups (P<0.05). There was no significant difference in effective rate and survival rate between the two groups (P<0.05). The effective rate of UCBT and BM/PB Allo-HSCT group was higher than that of ATG/ALG + CsA group (P<0.05). The effective rate of CsA ± androgen group was 37.5%, and that of supportive treatment group was the lowest (33.33%). There was no significant difference between UCBT group and BM/PB Allo-HSCT group and ATG/ALG group in quality of life scores, which were higher than CsA treatment group (P=0.000) and support treatment group (P=0.000). There was no significant difference in effective rate and quality of life between CsA ± androgen group and supportive treatment group. Summary/Conclusion: UCBT is comparable to bone marrow/peripheral blood Allo-HSCT in terms of effective rate, survival rate, basic cure rate and hematopoietic reconstruction rate, which is expected to become a treatment option for more SAA patients. UCBT is the best choice for young AA patients without HLA complete matched bone marrow transplantation (BMT).Keywords: Quality of life, Umbilical cord blood transplant, Clinical outcome, Aplastic anemia